HealthDay News — Research on an experimental treatment that combines transplanted neural stem cells with the protein 3K3A-APC shows it may be possible to reverse brain damage after stroke. The findings were published in Nature Medicine.

In mice, the protein triggered the stem cells to become functioning neurons. “We showed that 3K3A-APC helps the grafted stem cells convert into neurons and make structural and functional connections with the host’s nervous system,” senior author Berislav Zlokovic, MD, PhD, director of the Zilkha Neurogenetic Institute at the University of Southern California, said in a university news release. Zlokovic is also a scientific founder of ZZ Biotech, a company that is developing treatments with the protein used in this trial.

Zlokovic and his team now want to conduct a clinical trial to test whether this therapy is effective in stroke patients. If it’s successful, they plan to test the therapy in treating other neurological conditions, such as spinal cord injuries.

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In an ongoing clinical trial funded by the US National Institutes of Health, 3K3A-APC alone is being given to patients within a few hours of ischemic stroke, to determine if the protein can help protect against brain damage.

Disclosures: Dr Zlokovic is a founder of ZZ Biotech LLC, a biotechnology company with a mission to develop APC and its functional mutants for the treatment of stroke and other neurological disorders. Dr Griffin is a consultant for ZZ Biotech LLC and an inventor for some uses of 3K3A-APC.


Wang Y, Zhao Z, Rege SV, et al. 3K3A-activated protein C stimulates postischemic neuronal repair by human neural stem cells in mice. Nat Med. 2016; doi:10.1038/nm.4154.